EMA Backs Record Number of Drugs for Rare Diseases in 2014

Megan Brooks

January 09, 2015

The European Medicines Agency (EMA) gave positive opinions on a record number of orphan medicines for rare diseases in 2014.

Of a total of 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease, "providing therapies for patients who often have only few or no treatment options," the EMA notes in a news release.

For comparison, the agency recommended 11 orphan medicines in 2013, eight in 2012, and four each in 2011 and 2010.

2014 also saw positive opinions from the EMA on 40 new (nonorphan) drugs, three biosimilars, one drug for pediatric use, and 21 generic, hybrid, and informed consent applications.

Included among the orphan drugs is ataluren (Translarna, PTC Therapeutics), the first medicine for the treatment of Duchenne muscular dystrophy; afamelanotide (Scenesse, Clinuvel), a first for erythropoietic protoporphyria, a rare genetic disease that causes intolerance to light; and the stem cell product Holoclar (Chiesi Farmaceutici S.p.A.), the first-ever medicinal treatment for limbal stem cell deficiency, a rare eye condition that can result in blindness.

Special regulatory pathways were used for these three medicines: conditional marketing authorization for Translarna and Holoclar and approval under exceptional circumstances for Scenesse.

"These mechanisms are in place to potentially speed up market access for medicines that fulfill unmet medical needs but for which comprehensive data cannot be provided at the time of application for a marketing authorization," the EMA says.

New Active Substances

The agency also recommended eight cancer drugs in 2014, including four that target rare hard-to-treat cancers. They are olaparib (Lynparza, AstraZeneca) for BRCA-mutated ovarian cancer, ibrutinib (Imbruvica, Janssen) and obinutuzumab (Gazyvario, Roche) for chronic lymphocytic leukemia, and ramucirumab (Cyramza, Eli Lilly) for advanced gastric cancer or gastroesophageal junction adenocarcinoma.

"Overall, the number of medicines containing new active substances continues to increase," the EMA notes. "One in two medicines, either orphan or non-orphan, recommended for approval in 2014, contains a substance that has never been used in medicines before. These medicines have the potential to treat diseases for which no treatments were previously available or bring added benefit to patients over existing therapies," the agency says.

In 2014, the EMA saw an uptick in applicants seeking scientific support in the early stages of development. Nearly seven of 10 applicants received scientific advice from EMA's Committee for Medicinal Products for Human Use during the development phase of their product; this figure rises to four of five when it comes to innovative medicines.

"This is a significant increase compared with 2013 when only half of applicants who had a positive opinion for their medicine had received scientific advice," the EMA says.

The EMA granted seven positive opinions after an accelerated assessment, a pathway that aims to speed up the assessment of medicines that are expected to be of major benefit for public health, they say.

Four of the seven are for chronic hepatitis C: daclatasvir (Daklinza, Bristol-Myers Squibb), ledipasvir/sofosbuvir (Harvoni, Gilead), dasabuvir (Exviera, AbbVie), and ombitasvir + paritaprevir + ritonavir (Viekirax, AbbVie).

The other three are nintedanib (Ofev, Boehringer Ingelheim) for idiopathic pulmonary fibrosis, siltuximab (Sylvant, Janssen) for Castleman's disease, and ketoconazole (Ketoconazole HRA, Laboratoire HRA Pharma) for Cushing's syndrome.

In 2014, the CHMP granted a pediatric-use marketing authorization (PUMA) for Hemangiol (active ingredient propranolol; Pierre Fabre Dermatologie Laboratories) for the treatment of proliferating infantile hemangioma.

"PUMAs can be granted for medicines which are already authorized, but no longer under patent or supplementary certificate protection, and that have been developed specifically for children," the EMA explains. "As an incentive to stimulate the development of existing medicines for the treatment of children, PUMA medicines are granted 10 years of market protection."

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